Vertex Gets FDA's Priority Review For SNDA For Its ORKAMBI

Vertex Pharmaceuticals Incorporated VRTX revealed that the U.S. Food and Drug Administration has accepted its supplemental New Drug Application (sNDA) for priority review. According to the company, the review is for the use of ORKAMBI (lumacaftor/ivacaftor) in people with cystic fibrosis (CF) ages 6 to 11 who have two copies of the F508del mutation.

Vertex said that the regulator has approved its request for Priority Review of this sNDA, and a target review date of September 30, 2016 was set under the Prescription Drug User Fee Act (PDUFA).

The company's EVP and Chief Medical Officer, Jeffrey Chodakewitz, said, "As complications related to CF can occur early in life, we believe it is important to begin treatment as early as possible and this supplemental New Drug Application for approval of ORKAMBI in children as young as six is an important step in that direction. This submission demonstrates Vertex's continued progress toward our goal of developing medicines for all people with CF."

The pharmaceutical firm indicated that the sNDA was based on data from an open label Phase 3 clinical safety trial of ORKAMBI. Data from this trial would be presented at the 39th European Cystic Fibrosis Society conference to be held June 8 to 11 in Switzerland.

Vertex also disclosed that a six-moth final stage efficacy trial of children aged 6–11 was ongoing to support possible approval in the European Union. The company recently completed enrollment in this trial and, pending data from the study, plans to submit a Marketing Authorization Application (MAA) variation in the European Union in the first half of 2017 for children ages 6 to 11 who have two copies of the F508del mutation.

On Friday, the stock gained 1.33 percent.